News

Today, the Cystic Fibrosis Foundation announced that it awarded funding to enGene Inc. to develop a customized vehicle to deliver genetic-based therapies, such as gene therapy and gene editing, into the lung cells of people with cystic fibrosis (CF). Delivering genetic-based therapies to the lungs is a key hurdle to developing effective treatments for all people with CF, including individuals with two nonsense and rare mutations.

The Cystic Fibrosis Foundation awarded up to $14 million in additional funding to Synspira Therapeutics Inc. to develop a non-porcine enzyme replacement therapy as well as a treatment to help fight drug-resistant infections and reduce mucus thickness and inflammation. In 2017, the Foundation signed its first agreement with Synspira; with this new funding, the Foundation will have committed up to $22.55 million in total for the development of these drugs.

Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.

Corbus Pharmaceuticals reported today that the Phase 2b clinical trial of its potential anti-inflammatory drug lenabasum did not meet its primary objective of decreasing pulmonary exacerbations in people with cystic fibrosis.

The U.S. Food and Drug Administration has approved the use of Kalydeco^® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.

Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta^® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.